Crinetics to Present New Long-Term Data Demonstrating Durable Control of Once-Daily, Oral 笔础尝厂翱狈滨贵驰鈩� (Paltusotine) in Acromegaly at ENDO 2025
Crinetics Pharmaceuticals (Nasdaq: CRNX) announced new long-term data for 笔础尝厂翱狈滨贵驰鈩� (paltusotine), their investigational once-daily oral treatment for acromegaly, to be presented at ENDO 2025. The data comes from open-label extensions of two Phase 3 trials: PATHFNDR-1 and PATHFNDR-2.
Key findings through Week 96 show that PALSONIFY maintained stable IGF-1 levels in patients who switched from injectable treatments, with mean IGF-1 levels of 0.81 times the upper limit of normal. In PATHFNDR-2, patients showed sustained IGF-1 reductions, with a mean change of -0.81 脳 ULN at Week 84. The drug demonstrated durable biochemical control, reduced symptom burden, and was generally well-tolerated across both studies.
Crinetics Pharmaceuticals (Nasdaq: CRNX) ha annunciato nuovi dati a lungo termine su 笔础尝厂翱狈滨贵驰鈩� (paltusotine), il loro trattamento orale sperimentale una volta al giorno per l'acromegalia, che saranno presentati all'ENDO 2025. I dati provengono dalle estensioni in aperto di due studi di Fase 3: PATHFNDR-1 e PATHFNDR-2.
I risultati chiave fino alla settimana 96 mostrano che PALSONIFY ha mantenuto stabili i livelli di IGF-1 nei pazienti passati da trattamenti iniettabili, con livelli medi di IGF-1 pari a 0,81 volte il limite superiore della norma. In PATHFNDR-2, i pazienti hanno evidenziato riduzioni sostenute di IGF-1, con una variazione media di -0,81 脳 ULN alla settimana 84. Il farmaco ha dimostrato un controllo biochimico duraturo, una riduzione del carico sintomatico ed 猫 stato generalmente ben tollerato in entrambi gli studi.
Crinetics Pharmaceuticals (Nasdaq: CRNX) anunci贸 nuevos datos a largo plazo sobre 笔础尝厂翱狈滨贵驰鈩� (paltusotine), su tratamiento oral experimental una vez al d铆a para la acromegalia, que se presentar谩n en ENDO 2025. Los datos provienen de extensiones de etiqueta abierta de dos ensayos de Fase 3: PATHFNDR-1 y PATHFNDR-2.
Los hallazgos clave hasta la semana 96 muestran que PALSONIFY mantuvo niveles estables de IGF-1 en pacientes que cambiaron de tratamientos inyectables, con niveles medios de IGF-1 de 0,81 veces el l铆mite superior normal. En PATHFNDR-2, los pacientes mostraron reducciones sostenidas de IGF-1, con un cambio medio de -0,81 脳 ULN en la semana 84. El medicamento demostr贸 un control bioqu铆mico duradero, reducci贸n de la carga de s铆ntomas y fue generalmente bien tolerado en ambos estudios.
Crinetics Pharmaceuticals (雮橃姢雼�: CRNX)電� acromegaly(毵愲嫧牍勲寑歃�) 旃橂毳� 鞙勴暅 鞁ろ棙鞝� 瓴疥惮鞖� 頃橂（ 1須� 氤奠毄 鞎诫鞚� 笔础尝厂翱狈滨贵驰鈩�(韺旐埇靻岉嫶)鞚� 鞛リ赴 雿办澊韯瓣皜 ENDO 2025鞐愳劀 氚滍憸霅� 鞓堨爼鞛勳潉 鞎岆牳鞀惦媹雼�. 鞚� 雿办澊韯半姅 霊� 臧滌潣 3靸� 鞛勳儊鞁滍棙鞚� PATHFNDR-1瓿� PATHFNDR-2鞚� 瓿店皽鞐办灔 鞐瓣惮鞐愳劀 雮橃槰 瓴冹瀰雼堧嫟.
96欤检皑旯岇鞚� 欤检殧 瓴瓣臣鞐� 霐半ゴ氅�, PALSONIFY電� 欤检偓 旃橂鞐愳劀 鞝勴櫂頃� 頇橃瀽霌れ潣 IGF-1 靾橃箻毳� 鞎堨爼鞝侅溂搿� 鞙犾頄堨溂氅�, 韽夑窢 IGF-1 靾橃箻電� 鞝曥儊 靸來暅靹犾潣 0.81氚办榾鞀惦媹雼�. PATHFNDR-2鞐愳劀電� 頇橃瀽霌れ澊 歆�靻嶌爜鞚� IGF-1 臧愳唽毳� 氤挫榾鞙茧┌, 84欤检皑鞐� 韽夑窢 氤�頇旊焿鞚� -0.81 脳 ULN鞚挫棃鞀惦媹雼�. 鞚� 鞎诫鞚� 歆�靻嶌爜鞚� 靸濏檾頃欖爜 臁办爤鞚� 鞛呾頄堦碃, 歃濎儊 攵�雼挫潉 欷勳榾鞙茧┌ 霊� 鞐瓣惮 氇憪鞐愳劀 鞝勲皹鞝侅溂搿� 雮挫暯靹膘澊 膦嬱晿鞀惦媹雼�.
Crinetics Pharmaceuticals (Nasdaq : CRNX) a annonc茅 de nouvelles donn茅es 脿 long terme concernant 笔础尝厂翱狈滨贵驰鈩� (paltusotine), leur traitement oral exp茅rimental une fois par jour pour l鈥檃crom茅galie, qui seront pr茅sent茅es lors de l鈥橢NDO 2025. Ces donn茅es proviennent des extensions en ouvert de deux essais de phase 3 : PATHFNDR-1 et PATHFNDR-2.
Les r茅sultats cl茅s jusqu鈥櫭� la semaine 96 montrent que PALSONIFY a maintenu des niveaux stables d鈥橧GF-1 chez les patients ayant chang茅 de traitements injectables, avec des niveaux moyens d鈥橧GF-1 脿 0,81 fois la limite sup茅rieure normale. Dans PATHFNDR-2, les patients ont pr茅sent茅 des r茅ductions soutenues d鈥橧GF-1, avec un changement moyen de -0,81 脳 ULN 脿 la semaine 84. Le m茅dicament a d茅montr茅 un contr么le biochimique durable, une r茅duction de la charge symptomatique et a 茅t茅 g茅n茅ralement bien tol茅r茅 dans les deux 茅tudes.
Crinetics Pharmaceuticals (Nasdaq: CRNX) k眉ndigte neue Langzeitdaten f眉r 笔础尝厂翱狈滨贵驰鈩� (Paltusotin) an, ihre experimentelle einmal t盲glich oral einzunehmende Behandlung f眉r Akromegalie, die auf der ENDO 2025 vorgestellt werden sollen. Die Daten stammen aus offenen Verl盲ngerungsstudien zweier Phase-3-Studien: PATHFNDR-1 und PATHFNDR-2.
Wesentliche Ergebnisse bis Woche 96 zeigen, dass PALSONIFY stabile IGF-1-Werte bei Patienten, die von injizierbaren Behandlungen wechselten, aufrechterhielt, mit durchschnittlichen IGF-1-Werten von 0,81-fach des oberen Normbereichs. In PATHFNDR-2 zeigten die Patienten anhaltende IGF-1-Reduktionen mit einer mittleren Ver盲nderung von -0,81 脳 ULN in Woche 84. Das Medikament zeigte eine dauerhafte biochemische Kontrolle, reduzierte Symptomlast und wurde in beiden Studien im Allgemeinen gut vertragen.
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Insights
Crinetics shows positive long-term data for oral acromegaly drug PALSONIFY ahead of FDA decision, demonstrating durable hormonal control and symptom improvements.
Crinetics Pharmaceuticals has presented compelling long-term data for their once-daily oral therapy PALSONIFY (paltusotine) for acromegaly at ENDO 2025. The open-label extension (OLE) results from both PATHFNDR-1 and PATHFNDR-2 trials reveal stable control of IGF-1 levels - the key biomarker for acromegaly management - through 96 and 84 weeks, respectively.
For patients transitioning from injectable somatostatin receptor ligands (SRLs) to oral PALSONIFY in PATHFNDR-1, IGF-1 levels remained well-controlled at 0.81 times the upper limit of normal at Week 96, compared to 0.93 at baseline. This demonstrates non-inferiority to injectables while offering the convenience of oral administration - a significant quality-of-life improvement for patients requiring lifelong therapy.
The PATHFNDR-2 data in biochemically uncontrolled patients shows even more clinical value, with substantial IGF-1 reductions (mean change -0.81 脳 ULN) maintained through Week 84 in patients previously on placebo. This suggests PALSONIFY may provide a viable option for the challenging population of patients inadequately controlled on current therapies.
Particularly noteworthy is the significant reduction in symptom flares - from over 30% of days on injectable SRLs to just 6.2% on stable PALSONIFY dosing (p<0.0001). This dramatic improvement in day-to-day symptom stability could substantially impact patient quality of life, as acromegaly symptoms like headache, joint pain, and fatigue can be debilitating.
With a consistent safety profile and high retention rates in both extension studies (91% and 97.2%), these data strengthen PALSONIFY's positioning as a potential first-line oral therapy for acromegaly. As its PDUFA date approaches, these findings suggest PALSONIFY could become the new standard of care, particularly for patients struggling with the burden of monthly injections or inadequate control on current therapies.
Data show that PALSONIFY was well tolerated and IGF-1 levels remained stably controlled during long-term open label extensions of PATHFNDR-1 and PATHFNDR-2 studies
Additional PALSONIFY presentations demonstrate reductions in patient-reported symptom burden, including both symptom severity and rates of symptom flares
SAN DIEGO, July 13, 2025 (GLOBE NEWSWIRE) -- (Nasdaq: CRNX) today announced new data from its clinical development program evaluating once-daily, oral investigational PALSONIFYTM (paltusotine) in acromegaly will be presented in several oral and poster presentations at the Endocrine Society鈥檚 Annual Meeting, ENDO 2025. Notably, open-label extension (OLE) data from both the pivotal PATHFNDR-1 and PATHFNDR-2 trials will be featured in two presentations, showing the long-term clinical profile of PALSONIFY in people with acromegaly.
鈥淲ith once-daily, oral PALSONIFY鈥檚 PDUFA date quickly approaching, we鈥檙e excited to see a growing body of data that continues to underscore its potential as the next generation of care for people living with acromegaly,鈥� said Scott Struthers, Ph.D., Founder and Chief Executive Officer of Crinetics. 鈥淏uilding on the remarkable Phase 3 trial results from the PATHFNDR studies that we鈥檝e presented at ENDO previously, the new long-term data we鈥檙e showcasing this year provide further evidence of PALSONIFY鈥檚 ability to deliver durable IGF-1 control, sustained improvements in patient symptom burden, and a consistent safety profile.鈥�
PATHFNDR-1 Open-Label Extension (OLE) Study
The PATHFNDR-1 Phase 3 trial enrolled adults with acromegaly who were biochemically controlled on monthly injectable somatostatin receptor ligands (SRLs). Following a 36-week randomized, placebo-controlled period, 53 of 58 participants (
- In patients who transitioned from injected SRLs to once-daily oral PALSONIFY, mean insulin-like growth factor 1 (IGF-1) levels remained stable with IGF-1 (mean 卤 SE) of 0.93 卤 0.22 at OLE baseline and 0.81 卤 0.21 times the upper limit of normal (ULN) at Week 96, demonstrating durable biochemical control over this time span.
- Growth hormone (GH) levels were also stable, with mean values of 1.0 卤 1.0 ng/mL at baseline and 1.1 卤 1.2 ng/mL at Week 96.
- Symptom control, as measured by the Acromegaly Symptom Diary (ASD)鈥攁 seven-item daily patient-reported outcome assessing core acromegaly symptoms such as headache, joint pain, sweating, fatigue, and soft tissue swelling鈥攁lso remained stable at Week 96.
- PALSONIFY was generally well tolerated.
These results will be included in an oral presentation titled 鈥淒isease Control in Patients With Acromegaly Switching From Injected Somatostatin Receptor Ligands to Once-Daily Oral Paltusotine: Interim Results of the PATHFNDR-1 Open-Label Extension,鈥� taking place July 13 from 2:45-3:00 PM PT.
PATHFNDR-2 OLE Study
The PATHFNDR-2 trial is a randomized, double-blind, placebo-controlled Phase 3 study evaluating once-daily oral PALSONIFY in adults with biochemically uncontrolled acromegaly (baseline IGF-1 > 1.3 脳 ULN). After a 24-week RC period, 103 of 106 completers (
- Patients who had received placebo during the RC phase experienced sustained reductions in IGF-1, with a mean change from baseline of 鈥�0.81 脳 ULN at Week 84 (n=39). Direct-to-OLE participants (n=9) showed similar reductions (鈥�0.75 脳 ULN), while those who had been treated with PALSONIFY during the RC phase (n=40) maintained control (mean change 鈥�0.01 脳 ULN).
- GH, ASD symptom scores and pituitary tumor size were durably controlled over the study period.
- PALSONIFY was generally well tolerated.
These results will be included in a poster presentation titled 鈥淥nce-Daily Oral Paltusotine in the Treatment of Patients with Biochemically Uncontrolled Acromegaly: Interim Results of the PATHFNDR-2 Open-Label Extension,鈥� taking place July 14, from 12:00 PM 鈥� 1:30 PM PT.
Additional data presented at ENDO 2025 highlighted the impact of PALSONIFY on symptom burden in acromegaly. A pooled analysis of ASD scores from PATHFNDR-1 and PATHFNDR-2 showed that greater proportions of patients treated with PALSONIFY experienced less acromegaly symptom burden compared to placebo, regardless of the magnitude of the symptom effect, treatment history, or state of biochemical disease control. In an analysis of PATHFNDR-1 data, biochemically controlled patients switching from injected SRLs to PALSONIFY saw a significant drop in day to day symptom exacerbations 鈥� from over
About 笔础尝厂翱狈滨贵驰鈩� (Paltusotine)听
Crinetics鈥� lead development candidate, PALSONIFY, is the first investigational once-daily, oral, selectively-targeted somatostatin receptor type 2 (SST2) nonpeptide agonist that has completed Phase 3 clinical development for acromegaly and is in Phase 3 clinical development for carcinoid syndrome associated with neuroendocrine tumors. It was designed to be a once-daily oral option for the control of acromegaly and carcinoid syndrome. In Phase 3 studies, once-daily, oral PALSONIFY maintained IGF-1 levels and symptom control in patients with acromegaly who were switched from monthly injectable medications (PATHFNDR-1) and rapidly decreased IGF-1 levels and symptom burden in medically untreated acromegaly patients (PATHFNDR-2). IGF-1 is the primary biomarker endocrinologists use to manage acromegaly patients. Results from a Phase 2 study in carcinoid syndrome demonstrated rapid and sustained reductions in flushing episodes and bowel movement frequency, which are the most common symptoms of carcinoid syndrome, leading to the initiation of a Phase 3 trial for control of carcinoid syndrome in patients with neuroendocrine tumors.听
About Crinetics Pharmaceuticals听
Crinetics Pharmaceuticals is a clinical stage pharmaceutical company focused on the discovery, development, and commercialization of novel therapeutics for endocrine diseases and endocrine-related tumors. Crinetics鈥� lead development candidate, PALSONIFY (paltusotine), is the first investigational once-daily, oral, selective somatostatin receptor type 2 (SST2) nonpeptide agonist that is in clinical development for acromegaly and carcinoid syndrome associated with neuroendocrine tumors. Atumelnant is currently in development for congenital adrenal hyperplasia and ACTH-dependent Cushing鈥檚 syndrome. All of the company鈥檚 drug candidates are orally delivered, small molecule, new chemical entities resulting from in-house drug discovery efforts, including additional discovery programs addressing a variety of endocrine conditions such as hyperparathyroidism, polycystic kidney disease, Graves鈥� disease (including thyroid eye disease), diabetes, obesity and GPCR-targeted oncology indications.
Forward-Looking Statements听
This press release contains forward-looking statements within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended. All statements other than statements of historical facts contained in this press release are forward-looking statements, including statements regarding the plans and timelines for the clinical development of atumelnant and PALSONIFY, including the therapeutic potential and clinical benefits or safety profile thereof; the expected timing of the PDUFA target action date for our NDA submission to the FDA and of a potential EMA decision for our MAA for PALSONIFY for the treatment or maintenance of treatment of acromegaly in the United States and other applicable jurisdictions, and the plans and timelines for the commercial launch PALSONIFY if approved; and the therapeutic potential for our development candidates. In some cases, you can identify forward-looking statements by terms such as 鈥渕ay,鈥� 鈥渨ill,鈥� 鈥渟hould,鈥� 鈥渆xpect,鈥� 鈥減lan,鈥� 鈥渁nticipate,鈥� 鈥渃ould,鈥� 鈥渋ntend,鈥� 鈥渢arget,鈥� 鈥減roject,鈥� 鈥渃ontemplates,鈥� 鈥渂elieves,鈥� 鈥渆stimates,鈥� 鈥減redicts,鈥� 鈥減otential,鈥� 鈥渦pcoming鈥� or 鈥渃ontinue鈥� or the negative of these terms or other similar expressions. These forward-looking statements speak only as of the date of this press release and are subject to a number of risks, uncertainties and assumptions, including, without limitation, initial or topline data that we report may change following completion or a more comprehensive review of the data related to the clinical studies and such data may not accurately reflect the complete results of a clinical study, and the FDA and other regulatory authorities may not agree with our interpretation of such results; geopolitical events may disrupt Crinetics鈥� business and that of the third parties on which it depends, including delaying or otherwise disrupting its clinical studies and preclinical studies, manufacturing and supply chain, or impairing employee productivity; the success of Crinetics鈥� clinical studies and nonclinical studies; regulatory developments in the United States and foreign countries; clinical studies and preclinical studies may not proceed at the time or in the manner expected, or at all; the timing and outcome of research, development and regulatory review is uncertain, and Crinetics鈥� drug candidates may not advance in development or be approved for marketing; and the other risks and uncertainties described in the Company鈥檚 periodic filings with the Securities and Exchange Commission (SEC). The events and circumstances reflected in the company鈥檚 forward-looking statements may not be achieved or occur and actual results could differ materially from those projected in the forward-looking statements. Additional information on risks facing Crinetics can be found under the heading 鈥淩isk Factors鈥� in Crinetics鈥� periodic filings with the SEC, including its annual report on Form 10-K for the year ended December 31, 2024 and quarterly report on Form 10-Q for the quarter ended March 31, 2025. You are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date hereof. Except as required by applicable law, Crinetics does not plan to publicly update or revise any forward-looking statements contained herein, whether as a result of any new information, future events, changed circumstances or otherwise.
Contact:鈥�
惭别诲颈补:鈥�
Natalie Badillo鈥�
Head of Corporate Communications
(858) 345-6075
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Gayathri Diwakar
Head of Investor Relations
(858) 345-6340
FAQ
What are the key findings from Crinetics' PALSONIFY long-term data for acromegaly treatment?
How effective is PALSONIFY compared to injectable acromegaly treatments?
What was the patient retention rate in CRNX's PALSONIFY clinical trials?
What are the main advantages of Crinetics' PALSONIFY for acromegaly patients?
When will the clinical trial results for CRNX's PALSONIFY be presented?
